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Segment Information
 6 Months Ended
Jun. 30, 2025
Segment Reporting [Abstract]  
Segment Information Segment Information
Operating segments are defined as components of an entity for which separate financial information is available and that is regularly reviewed by the CODMs in deciding how to allocate resources and in assessing performance. The Company’s CODMs are (i) the Chief Executive Officer and (ii) the President and Chief Financial Officer. The Company is a clinical stage biotechnology company that operates as a single operating segment and has one reportable segment. In addition to reviewing the expenses in the consolidated statement of operations, the CODMs are provided with research and development costs for Rett syndrome, Batten disease, Early Discovery and Discontinued Programs, as well as categorized general and administrative expenses. The research and development programs are considered significant by the Company. The CODM assesses the financial performance of the segment and decides how to allocate resources based on net loss on a consolidated basis. The measure of segment assets is reported on the condensed consolidated balance sheets as total consolidated assets. The CODMs also assess performance of the segment according to preclinical and clinical data, the stage of development of the research and development programs, along with program specific expenses and market conditions in the pharmaceutical and biotechnology sectors.
As of June 30, 2025 and 2024, the Company did not have any significant long-lived assets located outside of the United States. Information on the segment and reconciliation to net loss is as follows (in thousands):
Three Months Ended June 30,Six Months Ended
June 30,
2025202420252024
Revenue under licensing agreements$— $925 $— $925 
Program specific expenses:
Rett syndrome$7,052 $2,798 $11,576 $4,817 
Batten disease401 1,734 1,246 2,924 
Early Discovery720 1,705 2,007 3,171 
Unallocated internal expenses:
Personnel-related5,125 4,557 10,529 9,275 
Stock-based compensation1,575 1,375 3,146 1,939 
Manufacturing3,635 2,815 6,742 5,794 
Other (a)
858 760 1,885 1,365 
Total research and development expenses$19,366 $15,744 $37,131 $29,285 
General and administrative specific expenses:
Personnel-related$2,212 $1,964 $4,696 $4,065 
Stock-based compensation1,833 939 4,310 1,419 
Professional and consultant fees970 998 2,247 2,500 
Office-related620 631 1,252 1,235 
Other (b)
1,080 783 2,364 1,334 
Total general and administrative expenses$6,715 $5,315 $14,869 $10,553 
Other income(c)
4,065 1,642 7,337 3,500 
Net loss$(22,016)$(18,492)$(44,663)$(35,413)
(a) The Other expense segment items category within research and development expense is mainly comprised of: Consultant fees for programs not specified above, IT Software and network support and rent expenses.
(b) The Other expense segment items category within general and administrative expense is mainly comprised of: Insurance, IT software and network support, market research, and tax expenses.
(c) Other income included in net loss is mainly comprised of: Interest income, interest expense, other income and other expense.